CRISPR-Cas9 editing of a TNPO3 mutation in a muscle cell model of limb-girdle muscular dystrophy type D2

A single-nucleotide deletion in the stop codon of the nuclear import receptor transportin-3 (TNPO3), also involved in human immunodeficiency virus type 1 (HIV-1) infection, causes the ultrarare autosomal dominant disease limb-girdle muscular dystrophy D2 (LGMDD2) by extending the wild-type protein....

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Detalles Bibliográficos
Autores principales: Poyatos-García, Javier, Blázquez-Bernal, Águeda, Selva-Giménez, Marta, Bargiela, Ariadna, Espinosa-Espinosa, Jorge, Vázquez-Manrique, Rafael P., Bigot, Anne, Artero, Ruben, Vilchez, Juan Jesús
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9898580/
https://www.ncbi.nlm.nih.gov/pubmed/36789274
http://dx.doi.org/10.1016/j.omtn.2023.01.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9898580/
https://www.ncbi.nlm.nih.gov/pubmed/36789274
http://dx.doi.org/10.1016/j.omtn.2023.01.004

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